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Wilson Therapeutics (publ) today announced that the first patient has been enrolled in the pivotal Phase 3 FOCuS clinical trial evaluating WTX101 (bis-choline tetrathiomolybdate), an investigational first-in-class copper-protein-binding agent with a unique mechanism of action, for the treatment of Wilson Disease. FOCuS is a randomized, controlled, rater-blinded, multi-center study that will enroll approximately 100 Wilson Disease patients, aged 18 years or over, to receive once-daily WTX101 or standard of care. The primary endpoint will be copper control assessed as the percentage change in free copper levels in blood from baseline to 48 weeks. Top-line data from the study is expected to be released H2 2019.

Carl Bjartmar, MD, PhD, Chief Medical Officer, Wilson Therapeutics commented: “The start of the FOCuS study represents an important milestone for Wilson Therapeutics as well as for patients and families affected by Wilson Disease, as WTX101 has the potential to become the first new medicine for this serious disorder in several decades. Through its novel mode of action with its high affinity and specificity to copper, WTX101 detoxifies excess copper in the liver and in the blood by forming stable tripartite complexes with copper and albumin that are then cleared through bile, the natural elimination route of copper. This unique approach to copper control has the potential to improve symptoms and associated disabilities in Wilson Disease patients, which was demonstrated in our successful Phase 2 trial. The Phase 3 FOCuS study is the first randomized controlled trial ever conducted to support approval of a new treatment option for Wilson Disease and we look forward to further evaluating the differentiated profile of WTX101 in this head-to-head study versus standard of care.”

The first patient was enrolled at the University of Michigan. Frederick K. Askari, MD, PhD, Associate Professor and Director of the Wilson Disease program at the University of Michigan, added: “WTX101 has shown great promise and I am excited to be part of the team advancing it through the clinic. The profile of this investigational drug is very encouraging, particularly the rapid control of clinical symptoms in combination with the simple once-daily dosing regimen and its promising side effect profile which could help improve compliance to therapy and treatment outcomes as a result. WTX101’s unique potential to remove copper from the saturated copper stores in the liver is also very encouraging. If the results of the Phase 2 study are replicated in this Phase 3 trial and the product gains regulatory approval, I am confident WTX101 will have the potential to make a significant difference for the Wilson Disease community.”